Welcome to our dedicated page for Orchard Therapeutics plc news (Ticker: ORTX), a resource for investors and traders seeking the latest updates and insights on Orchard Therapeutics plc stock.
Orchard Therapeutics plc (ORTX) is a pioneering biotechnology company dedicated to transforming the lives of children with life-threatening rare diseases through advanced gene therapies. The company focuses on the development, manufacturing, and commercialization of ground-breaking hematopoietic stem cell (HSC) gene therapies, which hold the potential to provide one-time treatments that address the underlying causes of severe genetic disorders.
Founded in 2015, Orchard Therapeutics collaborates with prestigious research centers globally to harness the life-saving potential of gene therapy. The company’s leadership team and partners bring over a decade of experience in advancing therapies for orphan diseases.
Orchard’s flagship product, Libmeldy® (atidarsagene autotemcel), is designed for the treatment of early-onset metachromatic leukodystrophy (MLD), a rare metabolic disorder. Libmeldy has received approval from the European Commission and the UK Medicines and Healthcare products Regulatory Agency and is under Priority Review by the U.S. FDA with a decision expected by March 18, 2024. The therapy aims to halt or slow disease progression by restoring enzymatic function.
The company is also advancing OTL-203 for mucopolysaccharidosis type I Hurler’s syndrome (MPS-IH) and OTL-201 for mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome. These programs utilize the same HSC gene therapy platform as Libmeldy.
In a strategic move, Kyowa Kirin recently acquired Orchard Therapeutics, enhancing its portfolio with Orchard's clinically differentiated gene therapy platform. This acquisition is expected to drive the development of new treatments and support ongoing and future launches, including the continued growth of Libmeldy in Europe and potential new approvals in the U.S.
Orchard Therapeutics continues its mission to deliver curative gene therapies for severe diseases by translating promising early-stage research into approved treatments available to patients worldwide. The team remains committed to leveraging its extensive experience and cutting-edge technology to address unmet medical needs in rare and underserved conditions.
Orchard Therapeutics (ORTX) announced that seven presentations from its neurometabolic portfolio will be showcased at the SSIEM Annual Symposium in Freiburg, Germany, from August 30 to September 2, 2022. Highlights include an oral presentation on Libmeldy (atidarsagene autotemcel) covering its clinical development and initial treatment in Europe. Additional presentations will focus on newborn screening for metachromatic leukodystrophy (MLD) and interim results from the investigational therapy OTL-203 for MPS-IH. Libmeldy is the first approved therapy for early-onset MLD patients in the EU.
Orchard Therapeutics (Nasdaq: ORTX) reported Q2 2022 total product sales of $3.1 million, with year-to-date sales reaching $8.2 million. The company is progressing toward securing reimbursement for its gene therapy Libmeldy across several European markets. R&D expenses fell 22% from Q1, totaling $22.0 million in Q2. Despite an operating loss of $32.4 million, a net loss of $50.9 million, and currency loss from exchange rate fluctuations, Orchard ended Q2 with $170.9 million in cash, sufficient to fund operations into 2024.
Prime Medicine, a biotechnology firm focused on innovative genetic therapies, has announced key leadership appointments. Richard Brudnick joins as Chief Business Officer, Niamh Alix as Chief Human Resources Officer, and Fubao Wang, Ph.D., as Senior Vice President, Head of Regulatory. These new executives bring extensive experience in business development, human resources, and regulatory affairs, enhancing the company's capacity to advance its Prime Editing technology. This technology aims to address a broad range of genetic diseases, offering potential one-time curative options.
jCyte, Inc., a biotechnology firm focused on retinal degenerative diseases, has appointed Dr. Stewart Craig as its new Chief Technology Officer. Dr. Craig brings over 30 years of experience in drug product development, manufacturing, and regulatory affairs. His extensive background in cell and gene therapies will assist jCyte in transitioning its investigational treatment, jCell, for retinitis pigmentosa (RP), from clinical to commercial stages. jCell has received both FDA Regenerative Medicine Advanced Therapy and Orphan Drug designations.
Orchard Therapeutics (Nasdaq: ORTX) reported Q1 2022 product sales for Libmeldy at $5.1 million from two patients. The company expanded access and reimbursement in three major European markets, with a significant deal in Germany at a €2.475 million price. Orchard anticipates a U.S. BLA filing for OTL-200 for metachromatic leukodystrophy by early 2023. R&D expenses surged to $28.2 million, while net loss reached $44.3 million. Cash reserves stand at $199 million, providing funding through 2024.
Orchard Therapeutics (Nasdaq: ORTX) announced seven key presentations at the ASGCT 25th Annual Meeting from May 16-19, 2022, in Washington, D.C. These include updated results on the OTL-203 clinical program for mucopolysaccharidosis type I Hurler syndrome and various preclinical findings on HSC gene therapy targeting neurodegenerative conditions, including frontotemporal dementia. Orchard's chief medical officer emphasized the progress in utilizing their HSC gene therapy platform to address significant medical challenges in gene therapy.
Orchard Therapeutics (Nasdaq: ORTX) will host a conference call and webcast on May 12, 2022, at 8:00 a.m. ET, to discuss business updates and first quarter financial results. The call can be accessed via a toll-free number or through their website. Orchard, a global leader in gene therapy, focuses on utilizing hematopoietic stem cell gene therapy to treat severe genetic diseases, and its diverse pipeline includes therapies developed from its acquisition of GSK's gene therapy portfolio.
Orchard Therapeutics (Nasdaq: ORTX) has secured an agreement with Gesetzliche Krankenversicherung Spitzenverband (GKV-SV) for reimbursed access to its gene therapy, Libmeldy®, for metachromatic leukodystrophy (MLD) patients in Germany. This follows a high therapeutic benefit rating from the Federal Joint Committee for early-onset MLD patients, making it the third European country to recognize the therapy. Orchard is also enhancing patient identification through newborn screening initiatives, addressing the rare, severe disease affecting approximately one in 100,000 live births.
Orchard Therapeutics (Nasdaq: ORTX) announced a significant reimbursement agreement with the Italian Medicines Agency (AIFA) for Libmeldy® (atidarsagene autotemcel), enabling access for all children with metachromatic leukodystrophy (MLD). This marks Orchard's second agreement in Europe this year to cover eligible MLD patients. Earlier, a patient from the Middle East received the therapy through international reimbursement in Italy. Libmeldy is the first approved therapy for early-onset MLD, addressing a critical unmet need in rare disease treatment.
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